A qualitative study on the impact of caring for an ambulatory individual with nonsense mutation Duchenne muscular dystrophy

A qualitative study on the impact of caring for an ambulatory individual with nonsense mutation Duchenne muscular dystrophy

Abstract Background Duchenne muscular dystrophy is a rare genetic neuromuscular disorder, which can result in early death due to disease progression. Ataluren is indicated for the treatment of nonsense mutation Duchenne muscular dystrophy, in ambulatory individuals aged two years and older. This stu...

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Bibliographic Details
Main Authors: Kate Williams, Ian Davidson, Mark Rance, Katharina Buesch, Sarah Acaster
Format: Article
Language:English
Published: SpringerOpen 2021-08-01
Series:Journal of Patient-Reported Outcomes
Subjects:
Duchenne muscular dystrophy
Nonsense mutation
Caregiver
Carer
Qualitative
Ataluren
Online Access:https://doi.org/10.1186/s41687-021-00344-8

Internet

https://doi.org/10.1186/s41687-021-00344-8

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